An Observational Study to Evaluate the Efficacy of Biweekly Romiplostim in Adult Patients With ITP

An Observational Study to Evaluate the Efficacy of Romiplostim Administered at Every Other Week in Adult Patients With Chronic Immune Thrombocytopenia (ITP) Who Attained Platelet Counts ≥ 50 x 109/L After Weekly Doses of Romiplostim

The aim of this study is to evaluate the efficacy of romiplostim administered at every other week in ITP patients who attained stable platelet counts ≥ 50 x 109/L for 4 consecutive weeks after weekly doses of romiplostim.

Study Overview

• Status: Unknown
• Conditions: Immune Thrombocytopenia
• Intervention / Treatment: Drug: Romiplostim

Detailed Description

Regarding the determination of appropriate dose, it is well known that romiplostim resulted in dose-dependent increases in platelet counts, but required dose for raising platelet count is variable between individuals according to each patient's ability to eliminate serum romiplostim. Because of this variability, after administration of initial 1mcg/kg of romiplostim, dose adjustment is recommended based on the platelet response to given dose of romiplostim. However, the optimal dose interval of romiplostim has rarely been studied. In pivotal studies, romiplostim was administered on a weekly schedule, and based on these studies, one dose at every week is being widely used. In one study in which ITP patients received weekly subcutaneous doses of romiplostim at a range from 3 to 15mcg/kg, half life of romiplostim was estimated from 1 to 34 days, suggesting that lengthening the interval between doses more than a week can be potentially possible in some patients.

• Study Type: Interventional
• Enrollment (Anticipated): 20
• Phase: Phase 2

Contacts and Locations

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Subject has an ability to provide written informed consent prior to participating to the study
2. Subject is male or female ≥ 18 years of age
3. Subject has a diagnosis of immune thrombocytopenia (ITP) per the American Society of Hematology guidelines.
4. Subject received at least 1 prior therapy for ITP including corticosteroids, immunoglobulin, or splenectomy, but had an insufficient response to any of these treatment (PLT count should be < 30x109/L before romiplostim initiation.)
5. If subject had ever received TPO receptor agonist before, the patient can participate this study only after 8 weeks' wash out period

Exclusion Criteria:

1. Subject has a history of hematological malignancy, myeloproliferative disorder, myelodysplastic syndrome (MDS), or bone marrow stem cell disorder
2. Subject has participated in any study evaluating PEG-rHuMGDF, recombinant human thrombopoietin (rHuTPO), or related platelet product within 8 weeks
3. Subject has a known hypersensitivity to any recombinant E coli-derived product
4. Subject has received any therapeutic drug or device that is not approved by the local regulatory health agency for any indication within 4 weeks of Screening
5. Subject is of reproductive potential and is not using adequate contraceptive precautions, in the judgment of the investigator
6. Subject is pregnant or breast feeding

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Romiplostim; Patients receiving romiplostim due to ITP	Drug: Romiplostim; Subcutaneous Romiplostim (weekly injection) -> if titrated with maintaining PLT count between 50-200 x 10^9/L -> Subcutaneous Romiplostim (biweekly injection); Other Names: Nplate

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy of biweekly romiplostim (platelet counts ≥ 30 x 10^9/L)	To evaluate the proportion of patients who had platelet counts ≥ 30 x 109/L during 4 or more weeks with biweekly romiplostim	up to 24 weeks

Collaborators and Investigators

• Sponsor: Samsung Medical Center
• Investigators: Principal Investigator: Jong Wook Lee, M.D., Seoul St. Mary's Hospital, The Catholic University of Korea

Study record dates

Study Major Dates

• Study Start: January 1, 2015
• Primary Completion (Anticipated): December 1, 2015
• Study Completion (Anticipated): January 1, 2016

Study Registration Dates

• First Submitted: January 6, 2015
• First Submitted That Met QC Criteria: January 11, 2015
• First Posted (Estimate): January 14, 2015

Study Record Updates

• Last Update Posted (Estimate): January 14, 2015
• Last Update Submitted That Met QC Criteria: January 11, 2015
• Last Verified: January 1, 2015

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Hemorrhage; Hemorrhagic Disorders; Blood Coagulation Disorders; Skin Manifestations; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia
• Other Study ID Numbers: NPT-I001